Recommendations concerning the information required to monitor nusinersen use in real-world settings

Notice Santécom: 126469

The recognized indication for nusinersen in the treatment of individuals with 5q spinal muscular atrophy (SMA) type I was modified in December 2018 to include presymptomatic individuals and those with type II or III of the disease, provided that clinical monitoring in a real-world setting is carried out.

INESSS thus determined in this guidance the minimum information required to monitor nusinersen use. The issued recommendations concern:

• the minimum information required for documenting the use of nusinersen in a real-world setting and the minimum frequency of information gathering;
• the conditions for implementing clinical monitoring.

The frequency at which health-care institutions should share the information and the measures to be taken to implement this clinical monitoring remain to be defined with the Ministère de la Santé et des Services sociaux. Work is in progress to this end.